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U of Florida and Penn ink licensing deal for gene therapy to treat retinal disease


By Jesse Schwartz
Published: June 13th, 2018

Biotech company Ophthotech Corporation has entered into an exclusive global license agreement with the University of Florida (UF) Research Foundation and the University of Pennsylvania (Penn) to commercialize a treatment for a disease that causes blindness.

The licensed technology is an adeno-associated virus (AAV) gene therapy to treat rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic disease characterized by progressive and severe loss of vision leading to blindness. Proof-of-concept studies of the therapy have shown promising results in a canine disease model, and Ophthotech plans to launch a Phase 1/2 clinical trial in early 2020.

“The scientific elegance of this novel gene therapy product is its design to knock down the expression of the mutant rhodopsin while delivering the replacement functional rhodopsin with a single AAV vector, restoring normal protein expression in preclinical studies,” says Kourous A. Rezaei, chief medical officer at Ophthotech.

“Collaborating with the eminent scientists at the University of Florida and the University of Pennsylvania reinforces Ophthotech’s commitment to build a gene therapy pipeline for treatment of retinal diseases based on cutting edge technology,” Rezaei says.

Under the agreement, Ophthotech will partner with scientists at UF and Penn to further develop the gene therapy.

“Orphan retinal degenerative diseases are potentially devastating to patients, who do not have any treatment options available to them,” says Alfred Lewin, a professor at UF. “Gene therapy presents a compelling scientific approach for treating and potentially curing a wide range of retinal diseases.”

Source: Business Wire

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